Amicus gets hit with an 'unexpected' setback on a gene therapy tapped to lead $600M SPAC spinout
Amicus has taken the axe and chopped a gene therapy for Batten disease out of the pipeline after an “unexpected” setback in the clinic.
In a corporate update on Monday, the biotech reported that its long-term extension data from a Phase I/II study revealed that the initial stabilization they had achieved in patients during the first 2 years wasn’t sustainable. So researchers are dropping the CLN6 Batten disease gene therapy program and turning to a CLN3 program, also for Batten disease, with a “higher dose, different promoter, and intra-cisterna magna route of delivery.”
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